UniQure's Huntington's Gene Therapy Shows Promising Results, Paving Way for Potential Approval

UniQure has reported groundbreaking results from its Phase 1/2 trial of AMT-130, a gene therapy for Huntington's disease, demonstrating a 75% slowing of disease progression after three years. This development positions the company to potentially launch the first genetic treatment for this devastating neurodegenerative disorder.

Landmark Clinical Trial Results

The pivotal Phase 1/2 trial evaluated 29 patients receiving either a low or high dose of AMT-130. Data from 12 patients who received the high dose and completed 36 months of follow-up showed statistically significant improvements compared to an external control group.

Key findings include:

• A 75% slowing of disease progression as measured by the composite Unified Huntington's Disease Rating Scale (cUHDRS)
• A 60% slowing in Total Functional Capacity (TFC) decline, a measure of patients' ability to maintain functional independence
• Reduction in cerebrospinal fluid neurofilament light (NfL) levels, a biomarker of neurodegeneration
• A manageable safety profile with AMT-130 being well-tolerated

Dr. Sarah Tabrizi, director of the University College London Huntington's Disease Center, emphasized the significance of these results, stating, "That means for one year of disease progression, they will have four years longer in terms of disease-free lives. It's a huge effect size, and will have massive effects on patients' lives."

Regulatory Path and Market Potential

UniQure plans to submit a Biologics License Application (BLA) to the FDA in the first quarter of 2026, with hopes for a potential U.S. launch later that year, pending approval. The company has already aligned with the FDA on key components of the statistical analysis plan and chemistry, manufacturing, and controls information to support the BLA through an accelerated pathway.

The announcement has had a significant impact on UniQure's market position:

• UniQure's stock price more than tripled, reaching levels not seen in over four years
• The company secured a debt refinancing deal with Hercules Capital, providing access to up to $125 million based on AMT-130's regulatory progress

Industry Impact and Future Outlook

AMT-130's success comes at a crucial time for the Huntington's disease treatment landscape, which has faced numerous setbacks in recent years. Several major pharmaceutical companies, including Roche, Wave Life Sciences, and Sage Therapeutics, have abandoned their Huntington's programs due to disappointing results.

UniQure's Chief Medical Officer, Dr. Walid Abi-Saab, expressed optimism about the therapy's potential: "These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington's disease, while also providing important evidence supporting one-time, precision-delivered gene therapies for the treatment of neurological disorders."

As UniQure prepares for regulatory discussions and a potential market launch, the pharmaceutical industry will be watching closely to see if AMT-130 can indeed become the first approved genetic treatment for Huntington's disease, potentially opening new avenues for gene therapy in neurodegenerative disorders.